Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

Rachael F. Grace, Christian Rosé, D. Mark Layton et al. · New England Journal of Medicine · 2019 · 159 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +13

Birth prevalence of disorders detectable through newborn screening by race/ethnicity

Lisa Feuchtbaum, Jennifer Carter, Sunaina Dowray et al. · Genetics in Medicine · 2012 · 148 citations · Full text

Public Health, Child Health, Pediatrics +14

AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency

Charles Kung, Jeff Hixon, Penelope A. Kosinski et al. · Blood · 2017 · 129 citations · Full text

Life Sciences, Biochemistry, Hematology +13

Mitapivat versus Placebo for Pyruvate Kinase Deficiency

Hanny Al‐Samkari, F. Galactéros, Andreas Glenthøj et al. · New England Journal of Medicine · 2022 · 94 citations · Full text

Medicine, Hematology, Pharmacology +13

The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model

Alessandro Mattè, Enrica Federti, Charles Kung et al. · Journal of Clinical Investigation · 2021 · 79 citations · Full text

Life Sciences, Hematology, Pharmacology +17